Clinical Trial With Riluzole in Spinocerebellar Ataxia Type 2 (ATRIL)

Last updated: June 18, 2021
Sponsor: Assistance Publique - Hôpitaux de Paris
Overall Status: Completed

Phase

3

Condition

Dyskinesias

Spinocerebellar Disorders

Friedreich's Ataxia

Treatment

N/A

Clinical Study ID

NCT03347344
P160927J
2017-001481-23
  • Ages > 18
  • All Genders

Study Summary

ATRIL is a multi-centric, double-blind randomized, two-arm controlled study. 42 SpinoCerebellar Ataxia type 2 (SCA2) patients, both gender, at least 18 years of age will be included.

Riluzole 50 mg will be administered (per os) twice a day, versus one group with placebo for 12 months.

Riluzole (Rilutek®) is a benzothiazole drug, market approved, for Amyotrophic Lateral Sclerosis (ALS). It delays the onset of ventilator-dependence or tracheostomy in selected patients and may increase survival.

Scale for the Assessment and Rating of Ataxia (SARA) will be used at M0, M6 and M12. To assess primary criterion, the percentage of patients with a decrease of at least 1 point of the SARA score between the inclusion visit, and Visit 3 (Months 12) will be calculated.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Genetically diagnosed SCA2 (CAG triplet in ATXN2 ≥ 33)
  • At least 18 years of age
  • Signature of informed consent
  • Covered by social security
  • SARA score ≥ 5 and ≤ 26
  • Age at onset ≤ 50 years old

Exclusion

Exclusion Criteria:

  • Treated with riluzole prior to the study
  • Hepatotoxic medication
  • Hypersensitivity to the active substance or to any of the excipients
  • Serious systemic illnesses or conditions known for enhancing the side effects ofriluzole
  • Contraindications for MRI examination
  • Participation in another therapeutic trial (3 months exclusion period)
  • Pregnancy or breastfeeding
  • Non abstinence or absence of effective contraception for women
  • Inability to understand information about the protocol
  • Persons deprived of their liberty by judicial or administrative decision
  • Adult subject under legal protection or unable to consent
  • Other ataxic syndromes than SCA2

Study Design

Total Participants: 42
Study Start date:
January 17, 2018
Estimated Completion Date:
December 14, 2020

Study Description

Inherited cerebellar ataxias are genetically heterogeneous neurological disorders. They are characterized by ataxic gait and cerebellar dysarthria that progresses over time with loss of ambulation and speech. The mutations by expansions of CAG triplets in the genes ATXN1 (SCA1), ATXN 2 (SCA2), 3 (SCA3), CACNA1A (SCA6), ATXN 7 (SCA7), and TBP (SCA17) are responsible for 50% of hereditary forms There is no curative or preventive treatment. This phase III study is a multi-centric, double-blind randomized, two-arm controlled study (one group with 50 mg Riluzole twice a day versus one group with placebo), to measure the efficacy of treatment with riluzole in SCA2 patients during 12 months. Amelioration is defined by a 1 point decrease of the SARA score.

Connect with a study center

  • Durr

    Paris, 75013
    France

    Site Not Available

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